Focused ultrasound-mediated brain genome editing

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Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions o...

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Detalles Bibliográficos
Autores principales: Leong, Kam, Lao, Yeh-Hsing, Ji, Robin, Zhou, Joyce, Snow, Kathy, Kwon, Nancy, Saville, Ethan, He, Siyu, Chauhan, Shradha, Chi, Chun-Wei, Datta, Malika, Zhang, Hairong, Quek, Chai Hoon, Cai, Sarah, Li, Mingqiang, Gaitan, Yaned, Bechtel, Lawrence, Wu, Shih-Ying, Lutz, Cathleen, Tomer, Raju, Murray, Stephen, Chavez, Alejandro, Konofagou, Elisa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Journal Experts 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9882596/
https://www.ncbi.nlm.nih.gov/pubmed/36712096
http://dx.doi.org/10.21203/rs.3.rs-2365576/v1
Descripción
Sumario:Gene editing in the mammalian brain has been challenging because of the restricted transport imposed by the blood-brain barrier (BBB). Current approaches rely on local injection to bypass the BBB. However, such administration is highly invasive and not amenable to treating certain delicate regions of the brain. We demonstrate a safe and effective gene editing technique by using focused ultrasound (FUS) to transiently open the BBB for the transport of intravenously delivered CRISPR/Cas9 machinery to the brain.

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